Soleno Therapeutics Announces Positive Outcome from Planned Data Safety Monitoring Board Review of Phase III DESTINY PWS Clinical Trial of DCCR in Prader-Willi Syndrome

March 14, 2019 Off By BusinessWire

REDWOOD CITY, Calif.–(BUSINESS WIRE)–Soleno Therapeutics, Inc. (NASDAQ: SLNO), a clinical-stage
biopharmaceutical company developing novel therapeutics for the
treatment of rare diseases, today announced that the Data Safety
Monitoring Board (DSMB) has recommended the continuation of the
Company’s Phase III trial in Prader-Willi Syndrome (PWS) patients,
DESTINY PWS, without any changes.

“We are delighted with the DSMB’s positive recommendation to continue
the Phase III trial as planned as it further supports DCCR’s safety
profile,” said Dr. Anish Bhatnagar, Chief Executive Officer of Soleno.
“We are continuing to enroll patients with 14 sites activated in DESTINY
PWS. In addition, patients continue to roll over into C602, the 9-month
open-label safety extension study for patients completing blinded
treatment in the DESTINY PWS study.”

The Phase III DESTINY PWS trial is a randomized, double-blind,
placebo-controlled study of once daily oral administration of DCCR
versus placebo in approximately 100 patients with a confirmed diagnosis
of PWS. The primary endpoint is change from baseline hyperphagia score
at Week 13. Patients who complete DESTINY PWS have the option to enroll
into C602.

The DSMB is a group of independent experts monitoring the safety of the
DESTINY PWS study. The DSMB reviews safety information and can make
recommendations to either continue the study without modification,
modify the study or terminate the study due to safety concerns.

In July 2018, the U.S. Food and Drug Administration designated the
investigation of DCCR for the treatment of PWS to be a Fast Track
development program. Prior to this, diazoxide choline received orphan
designation for the treatment of PWS in the U.S. and in the E.U.

For further information about the trial (NCT03440814), please visit: www.clinicaltrials.gov.

About PWS

The Prader-Willi Syndrome Association USA estimates that one in 12,000
to 15,000 people in the US have PWS. The hallmark symptom of this
disorder is hyperphagia, a chronic feeling of insatiable hunger that
severely diminishes the quality of life for PWS patients and their
families. Additional characteristics of PWS include behavioral problems,
cognitive disabilities, low muscle tone, short stature (when not treated
with growth hormone), the accumulation of excess body fat, developmental
delays, and incomplete sexual development. Hyperphagia can lead to
significant morbidities (e.g., stomach rupture, obesity, diabetes,
cardiovascular disease) and mortality (e.g., choking, accidental death
due to food seeking behavior). In a global survey conducted by the
Foundation for Prader-Willi Research, 96.5% of respondents (parent and
caregivers) rated hyperphagia as the most important or a very important
symptom to be relieved by a new medicine. There are currently no
approved therapies to treat the hyperphagia/appetite, metabolic,
cognitive function, or behavioral aspects of the disorder. Diazoxide
choline has received Orphan Drug Designation for the treatment of PWS in
the U.S. and E.U.

About Diazoxide Choline Controlled-Release Tablet

Diazoxide choline controlled-release tablet is a novel, proprietary
extended-release, crystalline salt formulation of diazoxide, which is
administered once-daily. The parent molecule, diazoxide, has been used
for decades in thousands of patients in a few rare diseases in neonates,
infants, children and adults, but has not been approved for use in PWS.
Soleno conceived of and established extensive patent protection on the
therapeutic use of diazoxide and DCCR in patients with PWS. The DCCR
development program is supported by data from five completed Phase I
clinical studies in healthy volunteers and three completed Phase II
clinical studies, one of which was in PWS patients. In the PWS Phase II
study, DCCR showed promise in addressing hyperphagia, the hallmark
symptom of PWS, as well as several other symptoms such as
aggressive/destructive behaviors, fat mass and abnormal lipid profiles.

About Soleno Therapeutics, Inc.

Soleno is focused on the development and commercialization of novel
therapeutics for the treatment of rare diseases. The company’s lead
candidate, DCCR, a once-daily oral tablet for the treatment of PWS, is
currently being evaluated in a Phase III clinical development program.
For more information, please visit www.soleno.life.

Forward-Looking Statements

This press release contains forward-looking statements that are subject
to many risks and uncertainties. Forward-looking statements include
statements regarding our intentions, beliefs, projections, outlook,
analyses or current expectations concerning, among other things, our
ability to complete the Phase III clinical development program of DCCR
in PWS in 2019. We may use terms such as “believes,” “estimates,”
“anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,”
“will,” “should,” “approximately” or other words that convey uncertainty
of future events or outcomes to identify these forward-looking
statements. Although we believe that we have a reasonable basis for each
forward-looking statement contained herein, we caution you that
forward-looking statements are not guarantees of future performance and
that our actual results of operations, financial condition and
liquidity, and the development of the industry in which we operate may
differ materially from the forward-looking statements contained in this
presentation. As a result of these factors, we cannot assure you that
the forward-looking statements in this press release will prove to be
accurate. Additional factors that could materially affect actual results
can be found in Soleno’s annual and quarterly reports filed with the
Securities and Exchange Commission, including under the caption titled
“Risk Factors.” Soleno expressly disclaims any intent or obligation to
update these forward-looking statements, except as required by law.